H. Lee Sweeney is an American scientist who studies

muscle Skeletal muscles (commonly referred to as muscles) are organs of the vertebrate muscular system and typically are attached by tendons to bones of a skeleton. The muscle cells of skeletal muscles are much longer than in the other types of muscl ...

Education and career

He received his undergraduate degree in biochemistry from

Massachusetts Institute of Technology The Massachusetts Institute of Technology (MIT) is a private land-grant research university in Cambridge, Massachusetts. Established in 1861, MIT has played a key role in the development of modern technology and science, and is one of the ...

in 1975 and his PhD from

Harvard Harvard University is a private Ivy League research university in Cambridge, Massachusetts. Founded in 1636 as Harvard College and named for its first benefactor, the Puritan clergyman John Harvard, it is the oldest institution of higher le ...

in physiology and biophysics in 1984. He then spent a year as research instructor in physiology at the University of Texas Southwestern Medical School, and then obtained an appointment as an assistant professor at the University of Texas at Austin. He joined the faculty of the

University of Pennsylvania The University of Pennsylvania (also known as Penn or UPenn) is a private research university in Philadelphia. It is the fourth-oldest institution of higher education in the United States and is ranked among the highest-regarded universitie ...

in 1989, became chair of the department of physiology in 1999, became founding director of the Paul D. Wellstone Muscular Dystrophy Cooperative Research Center in 2005. and eventually obtained an endowed chair and became director of the Penn Center for Orphan Disease Research and Therapy, which was founded in 2012. He left Penn and joined the faculty of

University of Florida The University of Florida (Florida or UF) is a public land-grant research university in Gainesville, Florida. It is a senior member of the State University System of Florida, traces its origins to 1853, and has operated continuously on its ...

College of Medicine in 2015, where he became the Thomas H. Maren M.D. Eminent Scholar Chair in Pharmacology and Therapeutics and became the director of the new Myology Institute there.

Research

In 1998 his lab published work in which they used

gene therapy Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. The first attempt at modifying human DN ...

to incorporate an extra copy of

IGF1 Insulin-like growth factor 1 (IGF-1), also called somatomedin C, is a hormone similar in molecular structure to insulin which plays an important role in childhood growth, and has anabolic effects in adults. IGF-1 is a protein that in humans is ...

into muscle cells of young and old mice; age-related muscle aging in the older mice was reversed and both old and mice became stronger. The paper received national press coverage, and the mice became known as "Schwarzenegger mice." He soon started receiving calls from athletes and coaches asking him to use this method for

gene doping Gene doping is the hypothetical non-therapeutic use of gene therapy by athletes in order to improve their performance in those sporting events which prohibit such applications of genetic modification technology, and for reasons other than the treat ...

; he was still receiving inquires as of 2008. He has written on the topic of gene doping in popular media and is often called upon to comment on the topic in the media. Sweeney collaborated with PTC Therapeutics in the discovery and development of

ataluren Ataluren, sold under the brand name Translarna, is a medication for the treatment of Duchenne muscular dystrophy. It was designed by PTC Therapeutics. Medical use Ataluren is used in the European Union to treat people with Duchenne muscular dys ...

. which was initially funded in part by Parent Project Muscular Dystrophy. Sweeney's lab had published work in 1999 showing that

gentamicin Gentamicin is an antibiotic used to treat several types of bacterial infections. This may include bone infections, endocarditis, pelvic inflammatory disease, meningitis, pneumonia, urinary tract infections, and sepsis among others. It is not e ...

could "cure" a

rodent model Rodents (from Latin , 'to gnaw') are mammals of the order Rodentia (), which are characterized by a single pair of continuously growing incisors in each of the upper and lower jaws. About 40% of all mammal species are rodents. They are na ...

Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy that primarily affects boys. Muscle weakness usually begins around the age of four, and worsens quickly. Muscle loss typically occurs first in the thighs and pelvis fol ...

and this led to the collaboration with PTC that produced ataluren—Sweeney was the last author on the initial publication of ataluren in ''Nature'' in 2007. Ataluren was approved to treat DMD in Europe in 2014. As of 2012 he had been an author on around 180 papers and reviews that had been cited around 16,000 times. Along with serving on the scientific advisory board of PTC, he does likewise with

Cytokinetics Cytokinetics, Inc. is a publicly traded biopharmaceutical company based in South San Francisco, California, that develops muscle activators and muscle inhibitors as potential treatments for people with diseases characterized by impaired or declin ...

and Solid Biosciences He is also on the scientific advisory board of the Parent Project Muscular Dystrophy.

References

{{DEFAULTSORT:Sweeney, Lee American physiologists Harvard University alumni University of Florida faculty Living people Massachusetts Institute of Technology School of Science alumni Year of birth missing (living people)